Study of Teduglutide in Japanese Participants With Short Bowel Syndrome

NCT ID: NCT03663582

Last Updated: 2020-08-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-07-06
• Study Completion Date: 2019-08-06

Brief Summary

The objectives of this clinical study are to evaluate the safety, efficacy, and pharmacokinetics (PK) of teduglutide in Japanese participants with short bowel syndrome (SBS) who are dependent on parenteral nutrition/intravenous (PN/IV) over a 24-week treatment period.

Conditions

Short Bowel Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Teduglutide 0.05 mg

Participants will receive teduglutide 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm for 24 weeks.

Group Type: EXPERIMENTAL

Teduglutide

Intervention Type: DRUG

Teduglutide 0.05 mg/kg SC injection will be administered once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm.

Syringe

Intervention Type: DEVICE

Teduglutide will be administered using syringe. Syringe is approved for use in Japan by Pharmaceuticals and Medical Devices Agency (PMDA).

Needle

Intervention Type: DEVICE

Teduglutide will be administered using needle. Needle is approved for use in Japan by PMDA.

Vial Adapter for Device

Intervention Type: DEVICE

Vial adapter for device is approved for use in Japan by PMDA.

Interventions

Teduglutide

Teduglutide 0.05 mg/kg SC injection will be administered once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm.

Intervention Type: DRUG

Syringe

Teduglutide will be administered using syringe. Syringe is approved for use in Japan by Pharmaceuticals and Medical Devices Agency (PMDA).

Intervention Type: DEVICE

Needle

Teduglutide will be administered using needle. Needle is approved for use in Japan by PMDA.

Intervention Type: DEVICE

Vial Adapter for Device

Vial adapter for device is approved for use in Japan by PMDA.

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

1. Ability to voluntarily provide written, signed, and informed consent to participate in the study.

2. Male or female 16 years of age or older at the time of signing informed consent.

3. Intestinal failure due to short bowel syndrome (SBS) as a result of major intestinal resection (example, due to injury, volvulus, vascular disease, cancer, Crohn's disease) that resulted in at least 12 continuous months of parenteral nutrition/intravenous (PN/IV) dependence at the time of informed consent.

4. Parenteral nutrition requirement of at least 3 times per week during the week before the screening visit and during the 2 weeks prior to the baseline visit.

5. Stable PN/IV requirement for at least 4 consecutive weeks immediately prior to the start of teduglutide treatment. Stability is defined as: a. Actual PN/IV usage is similar to prescribed PN/IV; b. Baseline (Visit 2) 48-hour oral fluid intake and urine output (I/O) volumes fall within +/- 25 percent (%) of the respective 48-hour I/O volumes at the last optimization visit; c. Urine output volume should NOT fall below 2 liter (L) and should not exceed 4 L per 48 hours at the last optimization visit, the stabilization visit, and the baseline visit.

6. For participants with a history of Crohn's disease, clinical remission for at least 12 weeks prior to the baseline visit as demonstrated by clinical assessment, which may include procedure-based evidence of remission.

7. Females of childbearing potential must agree to comply with the contraceptive requirements of the protocol.

8. An understanding, ability, and willingness to fully comply with study procedures and restrictions.

Exclusion Criteria

1. Participation in a clinical study using an experimental drug within 30 days or 5.5 halflives, whichever is longer, prior to screening, or concurrent participation in any other clinical study.

2. Use of glucagon-like peptide (GLP)-2 or human growth hormone or analogs of these hormones within the past 6 months.

3. Use of octreotide, GLP-1 analogs, dipeptidyl peptidase-IV inhibitors, or enteral glutamine within 30 days.

4. Previous use of teduglutide.

5. Participants with active inflammatory bowel disease (IBD) or participants with IBD who received a change in immunosuppressant therapy (example, azathioprine, anti- tumor necrosis factor (TNFs)) within the past 6 months.

6. Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, familial adenomatous polyposis, etc.

7. Chronic intestinal pseudo-obstruction or severe dysmotility.

8. Clinically significant intestinal stenosis or obstruction, or evidence of such on upper gastrointestinal (GI) series with small bowel follow-through, within the past 6 months.

9. Major GI surgical intervention, including bowel lengthening procedures, within the past 3 months (insertion of feeding tube or endoscopic procedure is allowed).

10. Unstable cardiac disease, (example, congestive heart failure, cyanotic disease, or congenital heart disease).

11. Moderate or severe renal impairment, defined as creatinine clearance less than (<) 50 millilitre (ml)/ minute (min).

12. Currently diagnosed with cancer or a history of any cancer except surgically curative skin cancer within the past 5 years.

13. Severe hepatobiliary disease including: a. Total bilirubin level greater than or equal to (>=) 2 times the upper limit of normal (ULN); b. Aspartate aminotransferase (AST) >=5 times ULN; c. Alanine aminotransferase (ALT) >=5 times ULN.

14. Active clinically significant pancreatic disease, including clinical signs of pancreatitis associated with elevations in serum amylase or lipase >=2 times ULN.

15. More than 4 SBS-related or PN/IV-related hospital admissions (example, central line associated bloodstream infection, bowel obstruction, severe fluid/electrolyte disturbances) within the past 12 months.

16. Unscheduled hospitalization within 30 days prior to screening.

17. Pregnant or lactating female.

18. Any condition or circumstance that in the investigator's opinion put the participant at any undue risk, prevent completion of the study, or interfere with analysis of the study results.

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Shire

Locations

Hiroshima University Hospital

Hiroshima, Hiroshima, Japan

Hyogo College of Medicine Hospital

Hyōgo, , Japan

Tohoku University Hospital

Miyagi-Ken, , Japan

Osaka University Hospital

Osaka, , Japan

Yokohama Municipal Citizen's Hospital

Yokohama, , Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

SHP633-306

Identifier Type: -

Identifier Source: org_study_id